I am a pediatric oncologist and currently acting chair of the Department of Pediatrics at the University of Massachusetts Medical School. I therefore have direct interactions with children and their families affected by life-threatening disease, as well as familiarity with a broader scope of pediatric treatment issues and clinical research.
I strongly support, both in general and specific, the recommendations of the American Academy of Pediatrics that the Pediatric Rule should be preserved and strengthened. More specifically:
· All components of the current 1998 Pediatric Rule must remain in place.
· Every pediatric study requested through BPCA or required by the Pediatric Rule must incorporate and reflect the spirit and intent of the ethical standards articulated in Subpart D.
· In assessing whether an adult indication occurs in children, FDA should consider the pathophysiology of disease in children vs adults, metabolic pathway, and mechanism of action of the drug to decide appropriateness and necessity of pediatric studies.
· There should be a single written request issued through BPCA that encompasses both off-label indications that need pediatric studies AND labeled indications that need pediatric studies.
This process is essential to the health of children, so that they might receive proper usage, dosing, and schedules for existing drugs and reap the benefits of modern pharmaceutical development of new drugs.